Metformin treatment in young children with fragile X syndrome

Hazel Maridith B. Biag, Laura A. Potter, Victoria Wilkins, Sumra Afzal, Alexis Rosvall, Maria Jimena Salcedo-Arellano, Akash Rajaratnam, Ramiro Manzano-Nunez, Andrea Schneider, Flora Tassone, Susan M. Rivera, Randi J. Hagerman

Research output: Contribution to journalArticle

1 Citation (Scopus)

Abstract

Background: Metformin is a drug commonly used in individuals with type 2 diabetes, obesity, and impaired glucose tolerance. It has a strong safety profile in both children and adults. Studies utilizing the Drosophila model and knock out mouse model of fragile X syndrome (FXS) have found metformin to rescue memory, social novelty deficits, and neuroanatomical abnormalities. These studies provided preliminary evidence that metformin could be used as a targeted treatment for the cognitive and behavioral problems associated with FXS. Previously, a case series of children and adults with FXS treated with metformin demonstrated improvements in irritability, social responsiveness, language, and hyperactivity. Methods: Here, we present nine children with FXS between 2 and 7 years of age who were treated clinically with metformin and monitored for behavioral and metabolic changes. Results: Parent reports and developmental testing before and after metformin are presented. There were improvements in language development and behavior (such as lethargy and stereotypy) in most of the patients. Conclusion: These results support the need for a controlled trial of metformin in children with FXS under 7 years old whose brains are in a critical developmental window and thus may experience a greater degree of clinical benefit from metformin.

Original languageEnglish (US)
Article numbere956
JournalMolecular Genetics and Genomic Medicine
Volume7
Issue number11
DOIs
StatePublished - Nov 1 2019

Fingerprint

Fragile X Syndrome
Metformin
Therapeutics
Lethargy
Language Development
Glucose Intolerance
Knockout Mice
Type 2 Diabetes Mellitus
Drosophila
Language
Obesity
Safety
Brain

Keywords

  • FMR1
  • fragile X syndrome
  • metformin
  • targeted treatments
  • translational medicine

ASJC Scopus subject areas

  • Molecular Biology
  • Genetics
  • Genetics(clinical)

Cite this

Biag, H. M. B., Potter, L. A., Wilkins, V., Afzal, S., Rosvall, A., Salcedo-Arellano, M. J., ... Hagerman, R. J. (2019). Metformin treatment in young children with fragile X syndrome. Molecular Genetics and Genomic Medicine, 7(11), [e956]. https://doi.org/10.1002/mgg3.956

Metformin treatment in young children with fragile X syndrome. / Biag, Hazel Maridith B.; Potter, Laura A.; Wilkins, Victoria; Afzal, Sumra; Rosvall, Alexis; Salcedo-Arellano, Maria Jimena; Rajaratnam, Akash; Manzano-Nunez, Ramiro; Schneider, Andrea; Tassone, Flora; Rivera, Susan M.; Hagerman, Randi J.

In: Molecular Genetics and Genomic Medicine, Vol. 7, No. 11, e956, 01.11.2019.

Research output: Contribution to journalArticle

Biag, HMB, Potter, LA, Wilkins, V, Afzal, S, Rosvall, A, Salcedo-Arellano, MJ, Rajaratnam, A, Manzano-Nunez, R, Schneider, A, Tassone, F, Rivera, SM & Hagerman, RJ 2019, 'Metformin treatment in young children with fragile X syndrome', Molecular Genetics and Genomic Medicine, vol. 7, no. 11, e956. https://doi.org/10.1002/mgg3.956
Biag HMB, Potter LA, Wilkins V, Afzal S, Rosvall A, Salcedo-Arellano MJ et al. Metformin treatment in young children with fragile X syndrome. Molecular Genetics and Genomic Medicine. 2019 Nov 1;7(11). e956. https://doi.org/10.1002/mgg3.956
Biag, Hazel Maridith B. ; Potter, Laura A. ; Wilkins, Victoria ; Afzal, Sumra ; Rosvall, Alexis ; Salcedo-Arellano, Maria Jimena ; Rajaratnam, Akash ; Manzano-Nunez, Ramiro ; Schneider, Andrea ; Tassone, Flora ; Rivera, Susan M. ; Hagerman, Randi J. / Metformin treatment in young children with fragile X syndrome. In: Molecular Genetics and Genomic Medicine. 2019 ; Vol. 7, No. 11.
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